A Phase II, Open Label, Extension Study to Assess the Effect of PRO044 in Patients With Duchenne Muscular Dystrophy
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 15
- 试验地点
- 5
- 主要终点
- Efficacy of PRO044 (composite of several measures)
研究概览
简要总结
The purpose of this study is to see whether PRO044 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 44 in the DNA for the dystrophin protein.
详细描述
A Phase II, open-label, extesion study. Following a Screening period of up to one month, subjects previously treated with PRO044, and eligible for enrolment in PRO044-CLIN-02, will be allocated to one of three groups to receive either 6 mg/kg or 9 mg/kg PRO044 weekly by IV infusion or 6 mg/kg weekly by SC injection for 48 weeks.
Safety and tolerability, pharmacokinetics (PK), pharmacodynamic (PD) and efficacy assessments will be conducted at regular intervals throughout the study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 9 Years 至 20 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Subjects previously treated with PRO
- •Continued use of glucocorticoids for a minimum of 60 days prior to study entry with a reasonable expectation that the subject will remain on steroids for the duration of the study. Changes to the dose regimen or cessation of glucocorticoids will be at the discretion of the Principle Investigator (PI) in consultation with the subject/parent and the Medical Monitor. If the subject is not on steroids, involvement in the study needs to be discussed with the medical monitor
排除标准
- •Current, or history of, liver or renal disease.
- •Acute illness within 4 weeks prior to the first dose of PRO044 (Week 1) which may interfere with the measurements.
- •Severe cardiac myopathy which in the opinion of the Investigator prohibits participation in this study
- •Need for daytime mechanical ventilation.
- •Screening aPTT above the upper limit of normal (ULN).
- •Screening platelet count below the lower limit of normal (LLN).
- •Use of anticoagulants, antithrombotics or antiplatelet agents.
- •Use of any investigational product within 6 months prior to the start of Screening for the study.
- •Current or history of drug and/or alcohol abuse.
研究组 & 干预措施
PRO044 SC 6 mg/kg
Weekly subcutaneous (SC) dosing with 6 mg/kg
干预措施: PRO044 SC 6 mg/kg (Drug)
PRO044 IV 6 mg/kg
Weekly intravenous (IV) dosing with 6 mg/kg
干预措施: PRO044 IV 6 mg/kg (Drug)
PRO044 SC 9 mg/kg
Weekly intravenous (IV) dosing with 9 mg/kg
干预措施: PRO044 IV 9 mg/kg (Drug)
结局指标
主要结局
Efficacy of PRO044 (composite of several measures)
时间窗: After 48 weeks of treatment
Efficacy parameters: Muscle Function * 6 Minute Walk Distance (6MWD) * North Star Ambulatory Assessment * Timed tests (10-meter walk/run, rising from floor, stair climb) * DMD Functional Outcomes Questionnaire (DMD-FOS) -for ambulant subjects only * Egen Klassification - for non-ambulant subjects. Muscle strength * Pulmonary Function (Spirometry) * Handheld myometry. Exploratory: * Performance Upper Limb (PUL). * Patient Reported Outcome measure (PROM).
Safety and tolerability of PRO044 (treatement emergent adverse events)
时间窗: After 48 weeks of treatment
Number of subjects with 1 or more treatement emergent adverse events following SC or IV PRO044 dosing
次要结局
- Assess the pharmacokinetics of PRO044 (composite of several measures)(After 48 weeks of treatment)